CA2986642A1 - Oligonucleotides antisens pour le traitement de l'epidermolyse bulleuse dystrophique - Google Patents
Oligonucleotides antisens pour le traitement de l'epidermolyse bulleuse dystrophique Download PDFInfo
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- CA2986642A1 CA2986642A1 CA2986642A CA2986642A CA2986642A1 CA 2986642 A1 CA2986642 A1 CA 2986642A1 CA 2986642 A CA2986642 A CA 2986642A CA 2986642 A CA2986642 A CA 2986642A CA 2986642 A1 CA2986642 A1 CA 2986642A1
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Classifications
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N15/00—Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
- C12N15/09—Recombinant DNA-technology
- C12N15/11—DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
- C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
-
- A—HUMAN NECESSITIES
- A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
- A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
- A61P17/00—Drugs for dermatological disorders
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- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/10—Type of nucleic acid
- C12N2310/11—Antisense
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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- C12N2310/00—Structure or type of the nucleic acid
- C12N2310/30—Chemical structure
- C12N2310/32—Chemical structure of the sugar
- C12N2310/321—2'-O-R Modification
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- C—CHEMISTRY; METALLURGY
- C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
- C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
- C12N2320/00—Applications; Uses
- C12N2320/30—Special therapeutic applications
- C12N2320/32—Special delivery means, e.g. tissue-specific
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- Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
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- Pharmacology & Pharmacy (AREA)
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- Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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Abstract
Un oligonucléotide antisens capable de prévenir ou de réduire l'inclusion d'exon 80 dans un ARNm COL7A1 humain, et des procédés permettant de prévenir ou de réduire l'inclusion d'exon 80 dans un ARNm COL7A1 humain.
Applications Claiming Priority (5)
| Application Number | Priority Date | Filing Date | Title |
|---|---|---|---|
| GBGB1508733.1A GB201508733D0 (en) | 2015-05-21 | 2015-05-21 | Antisense oligonucleotides |
| GB1508733.1 | 2015-05-21 | ||
| GB1516505.3 | 2015-09-17 | ||
| GBGB1516505.3A GB201516505D0 (en) | 2015-09-17 | 2015-09-17 | Antisense oligonucleotides |
| PCT/EP2016/061495 WO2016185041A1 (fr) | 2015-05-21 | 2016-05-20 | Oligonucléotides antisens pour le traitement de l'épidermolyse bulleuse dystrophique |
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| JP (2) | JP6871175B2 (fr) |
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| EA036393B1 (ru) | 2015-05-21 | 2020-11-05 | Вингс Терапьютикс, Инк. | Антисмысловые олигонуклеотиды для лечения дистрофического буллезного эпидермолиза |
| US10849917B2 (en) * | 2015-06-01 | 2020-12-01 | Sarepta Therapeutics, Inc. | Antisense-induced exon exclusion in type VII collagen |
| GB201902735D0 (en) | 2019-02-28 | 2019-04-17 | Proqr Therapeutics Ii Bv | Oligonucleotides for use in the treatment of dystrophic epidermolysis bullosa |
| WO2021113290A1 (fr) | 2019-12-03 | 2021-06-10 | Alamar Biosciences, Inc. | Dosage immunologique-sandwich lié à un acide nucléique (nulisa) |
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| US5139941A (en) | 1985-10-31 | 1992-08-18 | University Of Florida Research Foundation, Inc. | AAV transduction vectors |
| US5801154A (en) * | 1993-10-18 | 1998-09-01 | Isis Pharmaceuticals, Inc. | Antisense oligonucleotide modulation of multidrug resistance-associated protein |
| US5952221A (en) | 1996-03-06 | 1999-09-14 | Avigen, Inc. | Adeno-associated virus vectors comprising a first and second nucleic acid sequence |
| US20040023378A1 (en) * | 2002-07-31 | 2004-02-05 | Isis Pharmaceuticals Inc. | Antisense modulation of KIAA1531 protein expression |
| EP1857548A1 (fr) * | 2006-05-19 | 2007-11-21 | Academisch Ziekenhuis Leiden | Procédé et moyen permettant d'induire un saut d'exon |
| CN101896186A (zh) * | 2007-10-26 | 2010-11-24 | 莱顿教学医院 | 对抗肌肉病症的方式和方法 |
| CN102695797B (zh) * | 2009-06-16 | 2018-05-25 | 库尔纳公司 | 通过抑制针对胶原基因的天然反义转录物来治疗胶原基因相关的疾病 |
| ES2670495T3 (es) * | 2011-10-11 | 2018-05-30 | Inserm - Institut National De La Santé Et De La Recherche Médicale | Tratamiento de salto de exones para epidermólisis ampollosa distrófica |
| US9463244B2 (en) | 2013-03-15 | 2016-10-11 | Massachusetts Institute Of Technology | Compositions and methods for nucleic acid delivery |
| EA036393B1 (ru) | 2015-05-21 | 2020-11-05 | Вингс Терапьютикс, Инк. | Антисмысловые олигонуклеотиды для лечения дистрофического буллезного эпидермолиза |
| US10849917B2 (en) * | 2015-06-01 | 2020-12-01 | Sarepta Therapeutics, Inc. | Antisense-induced exon exclusion in type VII collagen |
| HUE051875T2 (hu) | 2015-06-15 | 2021-04-28 | Tirmed Pharma Ab | Egyláncú oligonukleotidok bõrrendellenességek orvosi kezelésében történõ alkalmazásra |
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2016
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- 2016-05-20 CA CA2986642A patent/CA2986642A1/fr active Pending
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- 2016-05-20 EP EP16724048.0A patent/EP3298144A1/fr active Pending
- 2016-05-20 US US15/575,559 patent/US10370660B2/en active Active
- 2016-05-20 KR KR1020177032446A patent/KR20180012255A/ko active IP Right Grant
- 2016-05-20 CN CN201680029306.7A patent/CN107667174B/zh active Active
- 2016-05-20 AU AU2016265457A patent/AU2016265457B2/en active Active
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- 2016-05-20 JP JP2017560600A patent/JP6871175B2/ja active Active
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2018
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- 2021-04-14 JP JP2021068721A patent/JP7188804B2/ja active Active
Also Published As
| Publication number | Publication date |
|---|---|
| EP3298144A1 (fr) | 2018-03-28 |
| AU2016265457B2 (en) | 2022-06-09 |
| EA036393B1 (ru) | 2020-11-05 |
| JP7188804B2 (ja) | 2022-12-13 |
| HK1245826A1 (zh) | 2018-08-31 |
| AU2016265457A1 (en) | 2017-12-07 |
| US20180245078A1 (en) | 2018-08-30 |
| JP2021112197A (ja) | 2021-08-05 |
| JP6871175B2 (ja) | 2021-05-12 |
| NZ737592A (en) | 2021-08-27 |
| WO2016185041A1 (fr) | 2016-11-24 |
| CN107667174A (zh) | 2018-02-06 |
| JP2018515122A (ja) | 2018-06-14 |
| KR20180012255A (ko) | 2018-02-05 |
| IL255180B2 (en) | 2023-11-01 |
| CN107667174B (zh) | 2021-10-26 |
| US20200024601A1 (en) | 2020-01-23 |
| US10370660B2 (en) | 2019-08-06 |
| IL255180A0 (en) | 2017-12-31 |
| IL255180B1 (en) | 2023-07-01 |
| EA201792406A1 (ru) | 2018-04-30 |
| MX2017014855A (es) | 2018-04-20 |
| US11104900B2 (en) | 2021-08-31 |
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